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HQK-1001

National Institutes of Health

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2,2-dimethylbutyric acid

Papers overview

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2014
2014
A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia.
To the editor: β-thalassemia syndromes comprise a global health burden. Hemoglobin E (HbE)–β thalassemia represents 60% of… 
2014
2014
A phase 2 study of HQK‐1001, an oral fetal haemoglobin inducer, in β‐thalassaemia intermedia
The β-thalassaemia syndromes represent a World Health Organization-designated global health burden (Weatherall et al, 2010… 
2013
2013
A Phase 2 Trial Of The Fetal Hemoglobin Inducer HQK-1001 (2,2-dimethylbutyrate, Sodium Salt) In Non-Transfusion-Dependent β-Thalassemia
Treatment of β-thalassemia is still largely dependent on supportive care, including blood transfusions and iron chelation. No… 
2012
2012
A phase 1/2 trial of HQK‐1001, an oral fetal globin inducer, in sickle cell disease
Therapeutics which reduce the pathology in sickle cell syndromes are needed, particularly noncytotoxic therapeutics. Fetal… 
2012
2012
A Phase 2 Trial of HQK-1001 (sodium 2,2-dimethylbutyrate), an Oral Fetal Globin Gene Inducer, in HbE-Beta Thalassemia in Thailand
Abstract 251 Beta thalassemia intermedia (BTI) syndromes are characterized by globin chain imbalance, ineffective erythropoiesis… 
Review
2012
Review
2012
A Randomized, Open-Label, Multicenter, Dose Escalation Study of HQK-1001 (2,2-Dimethylbutyrate, Sodium Salt) in Sickle Cell Disease
Abstract 998 Fetal hemoglobin (Hb F) induction is an effective therapeutic strategy in SCD. Widespread use of hydroxyurea (HU… 
Review
2011
Review
2011
A Phase 2 Clinical Study of HQK-1001 (2,2-dimethylbutyrate, sodium salt), a Fetal Hemoglobin Inducer, in Patients with Sickle Cell Disease
Abstract 1066 Fetal hemoglobin (Hb F) induction (anti-switching therapy) is an effective therapeutic strategy in sickle cell… 
2010
2010
Phase 1/2 Clinical Trial of HQK-1001, An Oral Fetal Hemoglobin Stimulant, In Sickle Cell Anemia
Abstract 943 Definitive therapies to reduce the underlying pathology in sickle cell syndromes are still needed, particularly non… 
2009
2009
HQK-1001 Has Additive HbF-Inducing Activity in Combination with Hydroxyurea and Decitabine.
Abstract 977 Several classes of HbF inducers, including chemotherapeutic agents, ESAs, and short chain fatty acids (SCFADs) have… 
2008
2008
Phase 1 Clinical Testing of HQK-1001, a Novel Oral Fetal Globin Gene Inducer
Development of non-cytotoxic, orally-active therapeutics, which induce fetal globin production, would be beneficial for treatment… 
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